U.S. Markets closed

Biohaven Enrolls First Patient in Phase 2/3 Trial of BHV-3500, Third-Generation CGRP Receptor Antagonist, for the Acute Treatment of Migraine

- BHV-3500 is the first CGRP receptor antagonist drug candidate to be administered in an intranasal (IN) formulation in late stage testing and is Biohaven's second drug candidate from its NOJECTION™ migraine platform

- Preliminary pharmacokinetics (PK) profile from Phase 1 clinical trial achieved targeted therapeutic exposures with significantly earlier time to maximal concentration (Tmax) than those observed with other small molecule CGRP receptor antagonists

- Topline efficacy and safety results expected 4Q2019

NEW HAVEN, Conn., April 1, 2019 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (BHVN), a biotechnology company focused on advancing innovative therapies for neurological and neuropsychiatric diseases, today announced that it enrolled the first patient in a Phase 2/3, double-blind, randomized, placebo-controlled, dose-ranging trial of intranasally administered BHV-3500 for the acute treatment of migraine. BHV-3500 is a novel, structurally distinct, third-generation calcitonin gene-related peptide (CGRP) receptor antagonist being developed by Biohaven.

Intranasal BHV-3500 is administered using the Aptar Pharma Unit Dose System (UDS) designed to enable systemic delivery of drugs. The device is approved with multiple drug products marketed in the United States using the Aptar technology. (Photo credit: Aptar Pharma)

In the trial, three doses of BHV-3500 (5, 10 and 20 mg) are being compared to placebo in the treatment of a migraine attack. With a planned enrollment of approximately 400 randomized subjects per treatment arm, the trial is powered to provide proof of efficacy on the regulatory endpoints of pain freedom and freedom from the most bothersome migraine-associated symptom at 2 hours post-dose. The trial is also designed to detect early onset of other clinical measures that are of great importance to patients, including pain relief and ability to return to normal functioning.

Vlad Coric, M.D., CEO of Biohaven, commented, "This is the first late-stage clinical trial designed to assess the efficacy of an intranasally administered CGRP receptor antagonist in the acute treatment of migraine. The PK profile from our recently completed Phase 1 trial suggests the potential for an ultra-rapid onset of action without the need for an injection. BHV-3500 is complementary to our lead migraine asset, rimegepant, which has met the primary efficacy endpoints in three completed Phase 3 clinical trials, with a favorable safety profile. We believe that intranasal BHV-3500 may provide people with ultra-rapid onset of migraine relief in a form that can easily be self-administered whenever and wherever a migraine strikes."

Intranasal BHV-3500 utilizes the Aptar Pharma Unit Dose System (UDS), which is designed to enable systemic delivery of drugs without the need for injection or administration by a healthcare professional. This device is FDA-approved to deliver multiple drug products marketed in the U.S., and is used by thousands of people every day. The Phase 1 trial used the Aptar Pharma UDS device and maintains important consistency of dosing throughout the BHV-3500 clinical development program. Catalent Pharma Solutions completes the fill/finish manufacturing of BHV-3500 and assembles the Aptar intranasal devices for Biohaven's clinical trials.

Robert Croop, M.D., Biohaven's Chief Development Officer – Neurology, added, "We are excited to advance BHV-3500, our third-generation CGRP receptor antagonist, into this Phase 2 trial to assess the best dose to move forward into a pivotal trial. The PK profile and intranasal administration differentiates BHV-3500 from other CGRP antagonists and may provide patients with an important new treatment option."

BHV-3500 is the second of Biohaven's CGRP receptor-targeting compounds to enter clinical trials. Biohaven's multiple CGRP receptor antagonist product candidates, including rimegepant, and expanded array of formulations including intranasal delivery and Catalent's Zydis® oral fast-dissolve tablet, are designed to meet patients' needs across the spectrum from acute to preventive treatment of migraine.

About BHV-3500

BHV-3500, the second product candidate in Biohaven's NOJECTION migraine platform, is a high affinity small molecule CGRP receptor antagonist with favorable potency and low protein binding. It is structurally distinct from rimegepant. The physicochemical properties of BHV-3500 make the drug candidate potentially suitable for multiple routes of delivery, including nasal, inhalation or oral administration. It is initially being developed as an intranasal formulation for the acute treatment of migraine.

About Migraine

Over 36 million Americans suffer from migraine. Acute attacks of migraine can differ in intensity and frequency, with many being highly disabling. More than 90 percent of migraine sufferers are unable to work or function normally during an attack. In the Global Burden of Disease Study, updated in 2015, migraine was ranked as the seventh highest cause worldwide of years lost due to disability. CGRP receptor antagonists represent a novel class of drug candidates for the treatment of migraine and are the first new class specific to the acute treatment of migraine in over 25 years. This unique and specific mode of action potentially offers an alternative to current agents, particularly for patients who have contraindications to the use of triptans, such as those with underlying cardiovascular diseases, or who either do not respond or have inadequate or inconsistent response to triptans or are intolerant to them.

About Biohaven

Biohaven is a clinical-stage biopharmaceutical company with a portfolio of innovative, late-stage product candidates targeting neurological diseases, including rare disorders. Biohaven has combined internal development and research with intellectual property licensed from companies and institutions including Bristol-Myers Squibb Company, AstraZeneca AB, Yale University, Catalent, Rutgers, ALS Biopharma LLC and Massachusetts General Hospital. Currently, Biohaven's lead development programs include multiple compounds across its CGRP receptor antagonist, glutamate modulation, and myeloperoxidase inhibitor platforms. Biohaven's common shares are listed on the New York Stock Exchange and traded under the ticker symbol BHVN. More information about Biohaven is available at www.biohavenpharma.com.

About Aptar Pharma

Aptar Pharma is part of AptarGroup, Inc., a leading global supplier of a broad range of innovative dispensing and sealing solutions for the beauty, personal care, home care, prescription drug, consumer health care, injectables, food and beverage markets. AptarGroup is headquartered in Crystal Lake, Illinois, with manufacturing facilities in North America, Europe, Asia and South America. For more information, visit aptar.com/pharma. Media Contact: Carolyn Penot, Aptar Pharma, +33 1 39 17 20 38, carolyn.penot@aptar.com

About Catalent

Catalent is the leading global provider of advanced delivery technologies and development solutions for drugs, biologics and consumer health products. With over 85 years serving the industry, Catalent has proven expertise in bringing more customer products to market faster, enhancing product performance and ensuring reliable clinical and commercial product supply. Catalent employs over 11,000 people, including over 1,800 scientists, at more than 30 facilities across five continents, and in fiscal 2018 generated approximately $2.5 billion in annual revenue. Zydis® is a registered trademark of Catalent. Catalent is headquartered in Somerset, New Jersey. For more information, visit www.catalent.com

Forward-Looking Statements

This news release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements involve substantial risks and uncertainties, including statements that are based on the current expectations and assumptions of the Company's management. All statements, other than statements of historical facts, included in this press release, including statements about the potential safety, efficacy and attractive mode of administration of BHV-3500 as a treatment for migraine, as well as its potential for ultra-rapid onset and sustained activity, the potential of the Company's CGRP receptor antagonist drug candidates to provide an improved, effective and safe treatment option for the acute and preventive treatment of migraine and the Company's expected timelines for receipt of data from clinical trials, are forward-looking statements. The use of certain words, including "believe," "potential" and "will" and similar expressions, is intended to identify forward-looking statements. The Company may not actually achieve the plans and objectives disclosed in the forward-looking statements, and you should not place undue reliance on the Company's forward-looking statements. Various important factors could cause actual results or events to differ materially from those that may be expressed or implied by our forward-looking statements, including those described in the "Risk Factors" section of the Company's Annual Report on Form 10-K filed with the Securities and Exchange Commission on February 28, 2019 and other filings Biohaven makes with the U.S. Securities and Exchange Commission from time to time. The forward-looking statements are made as of this date and the Company does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

For further information, contact Dr. Vlad Coric, Chief Executive Officer, Biohaven at Vlad.Coric@biohavenpharma.com

Biohaven Pharmaceuticals Logo (PRNewsfoto/Biohaven Pharmaceutical Holding)
Cision

View original content to download multimedia:http://www.prnewswire.com/news-releases/biohaven-enrolls-first-patient-in-phase-23-trial-of-bhv-3500-third-generation-cgrp-receptor-antagonist-for-the-acute-treatment-of-migraine-300821495.html